Novartis Itvisma Gains CHMP Boost for SMA Therapy

Basel, Switzerland | April 24, 2026

In a major breakthrough for rare disease treatment, Novartis has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency for its innovative gene therapy Itvisma®, targeting patients with 5q spinal muscular atrophy (SMA). This recommendation marks a pivotal step toward expanding access to advanced gene replacement therapies for older children, adolescents, and adults living with this debilitating neuromuscular condition.

Breakthrough Gene Therapy Targeting Root Cause of SMA

The investigational therapy Itvisma is designed as a one-time intrathecal gene replacement treatment, addressing the genetic root cause of SMA by delivering a functional copy of the SMN1 gene. Unlike traditional chronic therapies, this approach offers the potential to reduce long-term treatment burden and improve patient independence.

SMA is a rare genetic disorder characterized by the degeneration of motor neurons due to mutations in the SMN1 gene, leading to progressive muscle weakness and loss of essential functions such as breathing and movement. With a global prevalence of approximately 1–2 per 100,000 people, the disease represents a significant unmet medical need, particularly among older patients who historically had limited access to advanced therapies.

The CHMP’s positive opinion signals that Itvisma could become the first gene replacement therapy in the EU for patients aged two years and above, expanding beyond earlier treatments primarily focused on infants. This advancement reflects ongoing innovation in gene therapy and precision medicine, particularly in the field of neuromuscular disorders.

Clinical Evidence Demonstrates Significant Functional Gains

The recommendation is supported by robust clinical data from the Phase III STEER study, along with additional evidence from the STRENGTH and STRONG trials. In the pivotal STEER study, patients treated with Itvisma showed a statistically significant improvement of 2.39 points in the Hammersmith Functional Motor Scale (HFMSE) compared to 0.51 points in the control group, highlighting meaningful improvements in motor function over a 52-week period.

These results are particularly significant because even minor improvements in HFMSE scores can translate into tangible real-world benefits, such as improved mobility or the ability to perform daily activities independently. The therapy demonstrated consistent efficacy across both treatment-naïve and previously treated patients, reinforcing its broad therapeutic potential.

Safety profiles observed across studies were consistent, with adverse events comparable across clinical trials, supporting the therapy’s tolerability. Importantly, the single-dose administration model eliminates the need for repeated dosing, positioning Itvisma as a transformative option in long-term SMA management.

Regulatory Outlook and Future Impact on Rare Disease Care

Following the CHMP’s positive opinion, the European Commission is expected to deliver a final decision within approximately two months, which could pave the way for commercialization across the European Union. If approved, Itvisma would significantly expand treatment access and redefine care standards for SMA patients across multiple age groups.

Industry experts and patient advocacy groups have emphasized the importance of this development, noting that older SMA patients often face limited therapeutic options. The potential approval of Itvisma represents a critical step toward equitable access to cutting-edge therapies, addressing longstanding gaps in treatment availability.

From a strategic perspective, this milestone reinforces Novartis’ leadership in gene therapy innovation and neuroscience research, building on its existing portfolio in SMA and related disorders. The therapy also highlights the broader trend of shifting from symptomatic management to disease-modifying and curative approaches in rare diseases.

As regulatory processes advance, Itvisma stands poised to reshape the therapeutic landscape for spinal muscular atrophy, offering hope for improved quality of life, reduced healthcare burden, and long-term clinical benefits for patients worldwide.

Source: Novartis press release